Mini review – The role of Glucocerebrosidase and Progranulin as possible targets in the treatment of Parkinson's disease

• Published in: Revue neurologique 2021-11, Vol.177 (9), p.1082-1089
• Main Authors: Rodrigues, P.S., Kale, P.P.
• Format: Article
• Language: English
• Subjects: Acid ceramidase inhibitor; Ceramide; Gaucher disease; GBA1 gene; Glucocerebrosidase; Parkinson's disease; Progranulin; α-synuclein
• ISSN: 0035-3787
• DOI: 10.1016/j.neurol.2021.01.015

•The Gcase and PGRN need to be considered as possible targets in PD.•Use of treatment options considered for diseases linked with Gcase enzyme in PD.•Combination of carmofur and ambroxol treatment may benefit in parkinsonism.•Assess chaperone molecule, HDACis, acid ceramidase inhibitors, etc. in combination. As per recent reports, there is an association between glucocerebrosidase (Gcase) enzyme and Parkinson's disease (PD). In addition, certain mutations in the Gcase gene (GBA) and the progranulin (PGRN) gene are found to be linked with the imbalance in the levels of Gcase enzyme. This imbalance or decrease or impairment in Gcase activity can lead to Gaucher disease, frontotemporal lobar degeneration (FTLD), dementia, etc. Recent evidences suggest that the drugs used to treat these diseases can be used for PD. The present review has focused on the therapeutic approaches used for diseases linked with Gcase enzyme, which can be used for PD. The review also considered possible target specific novel strategies, which may help to meet the unmet needs in the treatment of PD.